Compassionate use policy

Compassionate use policy/expanded access policy

We receive a number of enquiries about access to our COMP360 investigational therapy.

We want to bring COMP360 psilocybin therapy to patients in a way that prioritises safety, efficacy and quality. To do this, we have to run a number of rigorous, large-scale clinical trials to generate robust data. Our priority now is to develop and conduct a larger phase III programme, a critical step before seeking regulatory approval.

We recognise there are patients who will not meet the criteria for our phase III trials. We are actively assessing which patient populations could be eligible for a compassionate use/expanded access programme, and we are working with regulatory authorities to determine the appropriate timing and conditions to offer pre-approval access to COMP360 psilocybin therapy.

More about compassionate use/expanded access

We conduct clinical trials to assess the safety and efficacy of investigational medicines which, if proven, will allow us to obtain the necessary approvals from regulatory authorities to provide patients with access to these medicines. Compassionate use is a treatment option that allows the use of an unauthorised medicine. This is also known as “expanded access”; COMPASS refers to it as compassionate use. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials.

Through the clinical development process, a drug is rigorously tested for safety and efficacy in humans. If proven, it is submitted to regulatory authorities for approval for use, and then made available through healthcare systems to as many patients in need as possible, as quickly as possible. Because we do not know during the clinical development whether the investigational medicine is safe or effective, compassionate use may present risks for both the patient and the clinical development programme. For patients, compassionate use may bring potential safety risks or a false sense that the medicine will provide benefit; for the clinical development programme, it can delay or jeopardise the approval of a new medicine sought by many.

We are responsible for ensuring the quality and integrity of our clinical trials and for minimising any risks to participants and possible future patients. When considering compassionate use of an investigational medicine, we consider many factors, such as the strength of the clinical data, the benefit-risk profile, the impact on the clinical development programme, the phase of development, and the probability and timing of regulatory approval.

A compassionate use programme can only be considered if a combination of the following conditions is met:

• There are no adequate alternative therapies or clinical trials available
• Sufficient preliminary efficacy and safety data exist for the therapy to enable COMPASS to make a benefit-risk analysis consistent with the establishment of a compassionate use programme
• Sufficient clinical data is available to identify an appropriate dose
• A patient’s treating physician and COMPASS both believe there is potential for the patient to reasonably expect benefit from the treatment, and there is robust evidence to support the possibility that the patient will benefit from it
• Adequate supply exists to support the ongoing clinical trials and approved compassionate use, until and if the product becomes commercially available
• The patient is not eligible for any of the COMPASS-sponsored studies of the treatment. Geographic limitations to participation in a trial would typically not mean a patient is ineligible
• Compassionate use will not adversely impact the clinical development programme, in particular, the conduct of a pivotal clinical trial that is required for regulatory approval
• The request must be made by the patient’s treating physician, unsolicited by COMPASS Pathways or any other individual or organisation

We will use the above criteria as we decide whether and when to offer compassionate use to COMP360 psilocybin therapy. We cannot, however, guarantee that a compassionate use programme will be offered; and should a programme become available, we currently cannot predict for which patients the investigational medicine could be made available.

Any pre-approval access to an investigational product must always comply with the applicable country-specific laws and regulations, including medicine importation requirements, and approvals must be secured from relevant regulatory bodies and the Institutional Review Board or Ethics Committee of the treating hospital.

If you have questions about our compassionate use programme, please contact us at info@compasspathways.com.